About Muscular Dystrophy

Muscular dystrophy is a neuromuscular, genetic disorder which results in the progressive deterioration of muscle strength and function. The most common form in childhood is Duchenne Muscular Dystrophy, which can see a young boy using a manual wheelchair by the age of 8, and being completely dependent on an electric one by his early teens. Life expectancy for this disorder is usually only until early adulthood.

There are many other types of neuromuscular disorders, affecting people of all ages. While most forms of muscular dystrophy occur in babies or children, some others appear in late adolescence or adulthood. The incidence of persons with a neuromuscular disorder is estimated to be 100 per 100,000 head of population. It is estimated that there are more than 20,000 people in Australia who have some form of neuromuscular disease.

Researchers and doctors are learning more about the causes of muscular dystrophy and various treatments are being trialled to stop the progression of the disease. Nonetheless, through early diagnosis, proper therapy and support an increased quality of life is possible.

Recently Prosensa provided reports on their financial results for the third quarter ending September 30, 2014 as well as an update on the regulatory plans and re-dosing for drisapersen, our lead exon-skipping drug candidate, and next steps for our follow-on exon skipping compounds for the treatment of Duchenne muscular dystrophy (DMD). Read More

The much anticipated worldwide survey to better understand healthcare for people with Duchenne muscular dystrophy - has just gone live. The Care-NMD survey will provide information about the current state of care for people with DMD all around Australia. This survey has already been completed in 19 countries. Read More

As we age, the cells responsible for helping to maintain, repair and build our muscle tissue are not exhausted. They are simply not being given the right instructions to carry out the work. Changing this communication problem could provide a way to treat muscular dystrophy. Read More